Pyridostigmine tends to improve subjective fatigability, but shows no significant effect on muscle strength

The SPACE clinical trial took place between 2016 and 2017 in the Netherlands. This single-centre, phase II, cross-over trial evaluated an 8-week course of pyridostigmine versus placebo in 35 people with SMA types II to IV.

The results, published in December 2022, show:

• no statistically significant difference in Nine-hole peg test (NHPT) and motor function measurement (MFM) results with pyridostigmine and placebo;
• good tolerance of pyridostigmine;
• a majority of participants (74.4%) reported a moderate or significant benefit of pyridostigmine on their fatigability (versus 29.7% on placebo).

 

Randomized double-blind placebo-controlled crossover trial with pyridostigmine in spinal muscular atrophy types 2-4. Stam M, Wijngaarde CA, Bartels B et al. Brain Commun. 2022 Dec 9;5(1):fcac324.